The primary outcome measure of this study is to evaluate and compare the incidence of adverse events in mitochondrial patients undergoing a diagnostic or therapeutic procedure between the three drug groups.

60

All

up to 17 Years (Child)

NCT02053766

HSC-MS-13-0600

January 2014

December 2018

December 2018

February 4, 2014

May 2, 2017

University of Texas Health Science Center at HoustonHouston, Texas, United States

Specific and generalized nutritional deficiencies in cohorts of IEM patients descriptive and functional immunologic data in cohorts of IEM patients by the collection of biologic specimens as above and the utilization of humanized mouse models.

250

All

2 Years and older (Child, Adult, Senior)

NCT01780168

13005313-HG-0053

January 29, 2013

December 1, 2022

December 1, 2022

January 30, 2013

December 8, 2017

National Institutes of Health Clinical Center, 9000 Rockville PikeBethesda, Maryland, United States